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Cannabinoid

Cannabidiol for Sanfilippo Syndrome

Phase 2 & 3

Waitlist Available

Research Sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up changes over 16 weeks of treatment versus 16 weeks of placebo

Summary

This trial aims to test the use of cannabidiol in treating Sanfilippo syndrome. They want to see if it is safe and effective in improving the behavior and daily functioning of patients with this

Who is the study for?

This trial is for individuals with Sanfilippo syndrome, a rare genetic condition. Participants will be selected based on their order of enrollment and must have caregivers able to complete various surveys about the patient's behavior and health throughout the study.

What is being tested?

The study tests cannabidiol (Epidiolex) against a placebo to assess its safety and effectiveness in treating neurobehavioral symptoms of Sanfilippo syndrome. It includes an initial safety cohort followed by a controlled group that switches between Epidiolex and placebo.

What are the potential side effects?

While specific side effects are not listed here, cannabidiol may commonly cause drowsiness, changes in appetite, diarrhea, fatigue, malaise, weakness or sleeping problems among others.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ changes over 16 weeks of treatment versus 16 weeks of placebo

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~changes over 16 weeks of treatment versus 16 weeks of placebo

This trial's timeline: 3 weeks for screening, Varies for treatment, and changes over 16 weeks of treatment versus 16 weeks of placebo for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Sanfilippo Behavior Rating Scale (SBRS)- mood/anger/aggression score

Side effects data

From 2022 Phase 1 & 2 trial • 3 Patients • NCT04238754

67%

Abdominal Pain

33%

Drowsiness

33%

Headache

33%

Nausea

100%

80%

60%

40%

20%

Study treatment Arm

Epidiolex

Placebo

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Epidiolex (cannabidiol)Experimental Treatment1 Intervention

Oral Epidiolex (cannabidiol) administered twice daily (BID) Week 1: 5 mg/kg/day BID Weeks 2-8: 10 mg/kg/day BID Weeks 9-16: 20 mg/kg/day BID 8-week washout; cross-over to placebo comparator starting on Week 24.

Group II: Placebo (PBO)Placebo Group1 Intervention

Week 1: Dose escalate to maximum tolerated placebo dose Weeks 2-16: Maximum tolerated placebo dose 8-week washout; cross-over to Experimental group starting at week 24, following the same dose-escalation as the Experimental arm.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Epidiolex

2023

Completed Phase 2

~530

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